High-Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT) in Light-Chain Deposition Disease (LCDD) and Immunoglobulin Deposition Disease (IGDD)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Hematologic Response Rate
研究概览
简要总结
RATIONALE: Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying the side effects of high-dose melphalan given together with stem cell transplant and to see how well it works in treating patients with immunoglobulin deposition disease or light-chain deposition disease.
详细描述
OBJECTIVES:
- To assess the tolerability of high-dose melphalan and autologous stem cell transplantation in patients with immunoglobulin deposition disease or light-chain deposition disease.
- To determine the hematologic response rate in patients treated with this regimen.
- To determine the predictability of early free light-chain response for heme response in patients treated with this regimen.
- To determine organ or clinical response in patients treated with this regimen.
- To determine overall survival of these patients.
OUTLINE:
- Stem cell mobilization: Patients undergo blood stem cell mobilization comprising filgrastim (G-CSF) subcutaneously once daily for 3 days (i.e., through the day before the last stem cell collection).
- Stem cell collection: Patients undergo collection of G-CSF-mobilized blood stem cells until the target number of stem cells (at least 2 x 10^6 cluster of differentiation-34-positive cells) is reached.
- Conditioning regimen: Patients receive high-dose melphalan IV on days -3 to -2.
- Autologous stem cell transplantation: Patients undergo blood stem cell infusion on day 0.
After completion of study therapy, patients are followed at 3, 6, and 12 months and then annually thereafter.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 120 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histologically confirmed light-chain deposition disease based on the following criteria:
- •Deposition of granular material containing free light-chain (FLC) immunoglobulins that did not bind Congo red
- •Evidence of a plasma cell dyscrasia, as defined by any of the following:
- •Monoclonal gammopathy in the serum or urine by immunofixation electrophoresis
- •Clonal plasmacytosis on bone marrow biopsy by immuno-histochemical
- •Elevated serum levels of FLC
- •Patients may enroll after stem cell collection (SCC) if all prestudy requirements are completed prior to starting SCC (i.e., ≥ 2.5 x 10^6 cells available for transplantation)
- •PRIOR CONCURRENT THERAPY:
- •Prior chemotherapy with alkylating agent allowed provided there is no evidence of myelodysplastic syndromes
- •Prior total dose of melphalan < 300 mg
- •More than 4 weeks since prior cytotoxic therapy and recovered
- •PATIENT CHARACTERISTICS:
- •Performance status 0-2
- •Left Ventricular Ejection Fraction (LVEF) ≥ 45% within the past 90 days
- •diffusing capacity of lung for carbon monoxide (DLCO) ≥ 50%
排除标准
- •No overt multiple myeloma, as defined by any of the following:
- •Greater than 30% bone marrow plasmacytosis
- •Extensive (i.e., > 2) lytic lesions
- •Hypercalcemia
- •No myocardial infarction, congestive heart failure, or arrhythmia refractory to therapy within the past 6 months
- •No prior malignancy except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, adequately treated stage I or II cancer from which the patient is currently in complete response, or any other cancer from which the patient has been disease-free for the past 5 years
- •No HIV positivity
研究组 & 干预措施
SCT with melphalan conditioning
Mobilization with Filgrastim Stem Cell Transplant Melphalan Conditioning Stem Cell infusion
干预措施: filgrastim (Biological)
SCT with melphalan conditioning
Mobilization with Filgrastim Stem Cell Transplant Melphalan Conditioning Stem Cell infusion
干预措施: melphalan (Drug)
SCT with melphalan conditioning
Mobilization with Filgrastim Stem Cell Transplant Melphalan Conditioning Stem Cell infusion
干预措施: Stem Cell Infusion (Procedure)
结局指标
主要结局
Hematologic Response Rate
时间窗: one year
次要结局
- Overall Survival(life)
- Tolerability(100 days)
- Organ or Clinical Response(One year)
- Predictability of Early Free Light-chain Response for Heme Response(One month)
研究者
Vaishali Sanchorawala
Principal Investigator
Boston Medical Center